Clinical Stage IV Cutaneous Melanoma AJCC v8Phase 1NCT05717140
Aerosolized Sargramostim Added to Immunotherapy for the Treatment of Patients With Metastatic Melanoma to the Lung
A Phase 1 study testing Aerosol Sargramostim, Biospecimen Collection and Computed Tomography in people with Clinical Stage IV Cutaneous Melanoma AJCC v8 and Metastatic Malignant Neoplasm in the Lung, run by Mayo Clinic at 1 site in United States.
Trial summary
This phase II trial tests the safety, best dose, and effectiveness of inhaled aerosolized sargramostim in combination with standard immunotherapy (nivolumab) for the treatment of patients with melanoma that has spread from where it first started (primary site) to the lung (metastatic to the lung). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Malignant Neoplasm in the Lung, Metastatic Melanoma
- What is being tested?
- Aerosol Sargramostim, Biospecimen Collection and Computed Tomography
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Oct 2025to 28 Jan 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >= 18 years
- Measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 as defined
- Confirmed diagnosis of unresectable stage IV metastatic melanoma involving the lungs (at least) that has progressed after at least ONE line of Food and Drug Administration (FDA) approved therapy (either immune checkpoint inhibitor or targeted therapy) OR relapse of melanoma following/during FDA-approved adjuvant or neo-adjuvant therapy
- Hemoglobin >= 8.0 g/dL (obtained =< 15 days prior to registration)
- Absolute neutrophil count (ANC) >= 1500/mm^3 (obtained =< 15 days prior to registration)
- Platelet count >= 75,000/mm^3 (obtained =< 15 days prior to registration)
Show all 17Show fewer
- Total bilirubin =< 1.5 x upper limit of normal (ULN) (obtained =< 15 days prior to registration)
- Alanine aminotransferase (ALT) AND aspartate aminotransferase (AST) =< 3.0 x ULN (obtained =< 15 days prior to registration)
- Serum creatinine =< 2.0 x ULN (obtained =< 15 days prior to registration)
- Calculated creatinine clearance >= 40 ml/min using the Cockcroft-Gault formula (obtained =< 15 days prior to registration)
- Pulse oximetry at rest > 90% without use of supplemental oxygen
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1
- Negative pregnancy test done =< 7 days prior to registration, for persons of childbearing potential only
- Persons able to become pregnant OR able to father a child must be willing to use an adequate method of contraception while on treatment and for 180 days (6 months) after last treatment dose on this study
- Provide written informed consent
- Willingness to provide mandatory blood specimens for correlative research
- Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)
Who cannot join
- Active pulmonary disease requiring ongoing medication
- NOTE: Stable chronic medication for asthma/chronic obstructive pulmonary disease (COPD) is allowed
- Metastatic uveal melanoma
- Any of the following because this study involves an investigational agent the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown:
- Pregnant persons
- Nursing persons
Show all 44Show fewer
- Persons of childbearing potential who are unwilling to employ adequate contraception
- Persons expecting to conceive or father children during the study or within 180 days (6 months) after the last treatment on this study
- Active central nervous system (CNS) metastases not previously treated
- NOTE: Patients with history of previously treated CNS metastases, not demonstrating evidence of progression for at least 8 weeks will be allowed
- NOTE: Patients with leptomeningeal metastases are not eligible
- Any of the following prior therapies:
- Allogeneic hematopoietic stem cell transplantation (HSCT)
- Solid organ transplantation
- Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- Immunocompromised patients and patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy
- NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial
- Active autoimmune disease requiring current systemic treatment with immunosuppressive or immune modulatory agents
- NOTE: Exceptions are allowed for the following conditions:
- Vitiligo
- Resolved childhood asthma/atopy
- Intermittent use of bronchodilators or inhaled steroids
- Daily steroids at dose of =< 10mg of prednisone (or equivalent)
- Local steroid injections
- Stable hypothyroidism on replacement therapy
- Stable diabetes mellitus on therapy (with or without insulin)
- Sjogren's syndrome
- Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) which is not considered a form of systemic treatment and is allowed
- Known uncontrolled intercurrent illness including, but not limited to:
- Ongoing or active infection requiring systemic therapy
- Serious, chronic gastrointestinal conditions associated with diarrhea (e.g., Crohn's disease or others)
- Symptomatic congestive heart failure
- Unstable angina pectoris
- Unstable cardiac arrhythmia or
- Psychiatric illness/social situations that would limit compliance with study requirements (e.g., known substance abuse)
- Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm
- History of severe hypersensitivity reactions to sargramostim or monoclonal antibodies
- Prior history of grade 4 immune related adverse event (irAE) with prior immune checkpoint inhibitor (ICI) therapy or failure to recover (< grade 1) from immune-related adverse event(s) from prior ICI therapy
- Failure to recover from adverse events related to any of the following therapies prior to registration:
- Chemotherapy
- Immunotherapy
- Targeted therapies (e.g., dabrafenib)
- Other investigational agents
- Radiation therapy
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: Aerosolized Granulocyte-Macrophage Colony Stimulating Factor (Sargramostim) in Combination With Anti-PD1 Therapy for the Treatment of Metastatic Melanoma to the Lung
This phase II trial tests the safety, best dose, and effectiveness of inhaled aerosolized sargramostim in combination with standard immunotherapy (nivolumab) for the treatment of patients with melanoma that has spread from where it first started (primary site) to the lung (metastatic to the lung). Sargramostim works to stimulate the immune system by prompting the bone marrow to produce more white blood cells. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. In this study, an aerosolized form of sargramostim is inhaled using a device called a nebulizer, which can deliver the drug directly to the lungs. Inhalation of aerosolized sargramostim in combination with nivolumab may be more effective at treating patients melanoma metastatic to the lung.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine feasibility, safety (maximum tolerated dose, MTD), and/or the maximally immunologically effective dose of aerosolized sargramostim (aSAR) administered as an inhalation in combination with nivolumab for the treatment of patients with metastatic malignant melanoma with evidence of pulmonary metastases.
SECONDARY OBJECTIVES:
- To assess the pharmacodynamics of inhaled aSAR administered in combination with nivolumab for the treatment of metastatic malignant melanoma.
II. To gather preliminary data on the clinical efficacy of aSAR + nivolumab therapy as 2nd line treatment for patients with metastatic melanoma.
OUTLINE: This is a dose escalation study of aerosolized sargramostim.
Patients receive aerosolized sargramostim via inhalation using the Aerogen Solo nebulization device and receive nivolumab intravenously (IV) on study. Patients also undergo collection of blood samples on study and undergo computed tomography (CT) or magnetic resonance imaging (MRI) at screening and on study.
- Conditions
- Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Malignant Neoplasm in the Lung, Metastatic Melanoma, Recurrent Melanoma, Unresectable Melanoma
- Being tested
- Aerosol Sargramostim, Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging, Medical Device Usage and Evaluation
- Medical subject headings
- Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aerosol SargramostimBiological
Also called Aerosol GM-CSF
Given via inhalation with nebulizer
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo collection of blood samples
Computed TomographyProcedure or surgery
Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography
Undergo CT
Magnetic Resonance ImagingProcedure or surgery
Also called Magnetic Resonance, Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR
Undergo MRI
Medical Device Usage and EvaluationOther
Use Aerogen Solo nebulization device
NivolumabBiological
Also called BMS-936558, CMAB819, MDX-1106, NIVO
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (aerosolized sargramostim, nivolumab) | Experimental | Patients receive aerosolized sargramostim via inhalation using the Aerogen Solo nebulization device and receive nivolumab IV on study. Patients also undergo collection of blood samples on study and undergo CT or MRI at screening and on study. Receives: Biological: Aerosol Sargramostim; Procedure: Biospecimen Collection; Procedure: Computed Tomography; Procedure: Magnetic Resonance Imaging; Other: Medical Device Usage and Evaluation; Biological: Nivolumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD)
The MRD is the dose level where at most 1 of the 6 patients treated at that dose level develops a dose-limiting event during the first cycle of treatment.
Measured: During the first cycle of treatment (each Cycle = 28 ±3 days)
Incidence of adverse events
Adverse events will be identified using Common Terminology Criteria for Adverse Events version 5.0. The maximum grade of each type of adverse event will be recorded for each patient. For each adverse event reported by dose level, the percentage of patients developing any degree of that adverse event as well as the percentage of patients developing a severe degree (grade 3 or higher) will be determined.
Measured: Up to 30 days after last treatment
Progression-free survival (PFS)
PFS distribution will be estimated using the Kaplan-Meier method.
Measured: Time from study entry to the documentation of disease progression, assessed up to 2 years after registration
Overall survival (OS)
OS distribution will be estimated using the Kaplan-Meier method.
Measured: Time from study entry to death, assessed up to 2 years after registration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2023
- Study start28 Oct 2025 (estimated)
- Primary completion28 Jan 2026 (estimated)
- Study completion28 Jan 2026 (estimated)
Why it stopped: Lack of funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Clinical Stage IV Cutaneous Melanoma AJCC v8Studies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Svetomir N. Markovic, M.D., Ph.D., Principal investigator, Mayo Clinic in Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05717140
- Sponsor's protocol ID
- MC210704
- Other IDs
- NCI-2023-00102, 22-002362
- Status last verified
- Aug 2025
- First submitted
- 20 Jan 2023
- Last updated
- 7 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Aerosolized Granulocyte-Macrophage Colony Stimulating Factor (Sargramostim) in Combination With Anti-PD1 Therapy for the Treatment of Metastatic Melanoma to the Lung
- ClinicalTrials.gov ID
- NCT05717140
- Acronym / sponsor ID
- MC210704
- Conditions
- Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Malignant Neoplasm in the Lung, Metastatic Melanoma, Recurrent Melanoma, Unresectable Melanoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 28 Oct 2025 (estimated)
- Primary completion
- 28 Jan 2026 (estimated)
- Study completion
- 28 Jan 2026 (estimated)
- First posted
- 8 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial tests the safety, best dose, and effectiveness of inhaled aerosolized sargramostim in combination with standard immunotherapy (nivolumab) for the treatment of patients with melanoma that has spread from where it first started (primary site) to the lung (metastatic to the lung). Sargramostim works to stimulate the immune system by prompting the bone marrow to produce more white blood cells. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. In this study, an aerosolized form of sargramostim is inhaled using a device called a nebulizer, which can deliver the drug directly to the lungs. Inhalation of aerosolized sargramostim in combination with nivolumab may be more effective at treating patients melanoma metastatic to the lung.
Detailed description
PRIMARY OBJECTIVE:
- To determine feasibility, safety (maximum tolerated dose, MTD), and/or the maximally immunologically effective dose of aerosolized sargramostim (aSAR) administered as an inhalation in combination with nivolumab for the treatment of patients with metastatic malignant melanoma with evidence of pulmonary metastases.
SECONDARY OBJECTIVES:
- To assess the pharmacodynamics of inhaled aSAR administered in combination with nivolumab for the treatment of metastatic malignant melanoma.
II. To gather preliminary data on the clinical efficacy of aSAR + nivolumab therapy as 2nd line treatment for patients with metastatic melanoma.
OUTLINE: This is a dose escalation study of aerosolized sargramostim.
Patients receive aerosolized sargramostim via inhalation using the Aerogen Solo nebulization device and receive nivolumab intravenously (IV) on study. Patients also undergo collection of blood samples on study and undergo computed tomography (CT) or magnetic resonance imaging (MRI) at screening and on study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (aerosolized sargramostim, nivolumab) | Experimental | Biological: Aerosol Sargramostim, Procedure: Biospecimen Collection, Procedure: Computed Tomography, Procedure: Magnetic Resonance Imaging, Other: Medical Device Usage and Evaluation, Biological: NivolumabPatients receive aerosolized sargramostim via inhalation using the Aerogen Solo nebulization device and receive nivolumab IV on study. Patients also undergo collection of blood samples on study and undergo CT or MRI at screening and on study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Age >= 18 years
- Measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 as defined
- Confirmed diagnosis of unresectable stage IV metastatic melanoma involving the lungs (at least) that has progressed after at least ONE line of Food and Drug Administration (FDA) approved therapy (either immune checkpoint inhibitor or targeted therapy) OR relapse of melanoma following/during FDA-approved adjuvant or neo-adjuvant therapy
- Hemoglobin >= 8.0 g/dL (obtained =< 15 days prior to registration)
- Absolute neutrophil count (ANC) >= 1500/mm^3 (obtained =< 15 days prior to registration)
Key exclusion criteria (44 listed)
- Active pulmonary disease requiring ongoing medication
- Metastatic uveal melanoma
- Any of the following because this study involves an investigational agent the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown:
- Active central nervous system (CNS) metastases not previously treated
- Any of the following prior therapies:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Aerosol SargramostimAlso: Aerosol GM-CSFGiven via inhalation with nebulizer | Biological | Treatment (aerosolized sargramostim, nivolumab) |
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Specimen CollectionUndergo collection of blood samples | Procedure or surgery | Treatment (aerosolized sargramostim, nivolumab) |
| Computed TomographyAlso: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial TomographyUndergo CT | Procedure or surgery | Treatment (aerosolized sargramostim, nivolumab) |
| Magnetic Resonance ImagingAlso: Magnetic Resonance, Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MRUndergo MRI | Procedure or surgery | Treatment (aerosolized sargramostim, nivolumab) |
| Medical Device Usage and EvaluationUse Aerogen Solo nebulization device | Other | Treatment (aerosolized sargramostim, nivolumab) |
| NivolumabAlso: BMS-936558, CMAB819, MDX-1106, NIVOGiven IV | Biological | Treatment (aerosolized sargramostim, nivolumab) |
Primary outcome
Maximum tolerated dose (MTD)
Time frame: During the first cycle of treatment (each Cycle = 28 ±3 days)
The MRD is the dose level where at most 1 of the 6 patients treated at that dose level develops a dose-limiting event during the first cycle of treatment.
Incidence of adverse events
Time frame: Up to 30 days after last treatment
Adverse events will be identified using Common Terminology Criteria for Adverse Events version 5.0. The maximum grade of each type of adverse event will be recorded for each patient. For each adverse event reported by dose level, the percentage of patients developing any degree of that adverse event as well as the percentage of patients developing a severe degree (grade 3 or higher) will be determined.
Progression-free survival (PFS)
Time frame: Time from study entry to the documentation of disease progression, assessed up to 2 years after registration
PFS distribution will be estimated using the Kaplan-Meier method.
Overall survival (OS)
Time frame: Time from study entry to death, assessed up to 2 years after registration
OS distribution will be estimated using the Kaplan-Meier method.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2025.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
Related trials for Clinical Stage IV Cutaneous Melanoma AJCC v8
RecruitingPhase 2
BiCaZO: A Study Combining Two Immunotherapies (Cabozantinib and Nivolumab) to Treat Patients With Advanced Melanoma or Squamous Cell Head and Neck Cancer, an immunoMATCH Pilot Study
Clinical Stage III Cutaneous Melanoma AJCC v8, Clinical Stage III HPV-Mediated (p16-Positive) Oropharyngeal Carcinoma AJCC v8
Active, not recruitingPhase 2
A Study to Compare the Administration of Pembrolizumab After Surgery Versus Administration Both Before and After Surgery for High-Risk Melanoma
Acral Lentiginous Melanoma, Clinical Stage III Cutaneous Melanoma AJCC v8
Active, not recruitingPhase 3
Dabrafenib and Trametinib Followed by Ipilimumab and Nivolumab or Ipilimumab and Nivolumab Followed by Dabrafenib and Trametinib in Treating Patients With Stage III-IV BRAFV600 Melanoma
Clinical Stage III Cutaneous Melanoma AJCC v8, Clinical Stage IV Cutaneous Melanoma AJCC v8
Active, not recruitingPhase 1
Tacrolimus, Nivolumab, and Ipilimumab in Treating Kidney Transplant Recipients With Selected Unresectable or Metastatic Cancers
Clinical Stage III Cutaneous Melanoma AJCC v8, Clinical Stage III Cutaneous Merkel Cell Carcinoma AJCC v8
Active, not recruitingPhase 1/2
Testing the Addition of the Anti-cancer Drug, Tazemetostat, to the Usual Treatment (Dabrafenib and Trametinib) for Metastatic Melanoma That Has Progressed on the Usual Treatment
Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Melanoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Svetomir N. Markovic, M.D., Ph.D. principal investigator
Mayo Clinic in Rochester
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Aerosol Sargramostim, Biospecimen Collection and Computed Tomography in people with Clinical Stage IV Cutaneous Melanoma AJCC v8 and Metastatic Malignant Neoplasm in the Lung, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 44 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 7 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) (time frame: During the first cycle of treatment (each Cycle = 28 ±3 days)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2025; AzTrials last refreshed its copy on 8 Nov 2026.